Patients with serious and/or life-threatening conditions can receive compassionate use access to promising treatments when they are not able to participate in a clinical trial and have no comparable or effective therapeutic options. The number of patients receiving access through these mechanisms remains very low. Biopharmaceutical companies can benefit from these programs, where evidence has been successfully used to support over 38 new drug applications and marketing approvals by the U.S. FDA. Anova today published a White Paper discussing the real world value for biopharmaceutical companies participating in compassionate use programs.
Many issues arise when patients facing serious and/or life-threating disease advocate for broadened use of compassionate use pathways. Most have either been demonstrated to be false (e.g., increased availability of investigational products through compassionate use threatens traditional drug development) or are easily addressed by the application of technology and careful planning (e.g., reduction in time and cost to receive, process and approve requests and the collection of quality data).
Biopharmaceutical companies should be incentivized to create robust compassionate use programs to provide promising new therapies to patients with serious and/or immediately life-threatening disease. Clinical development units should think about compassionate use when new treatments complete Phase I clinical testing. Whenever possible, resources should be developed to support these programs and when appropriate, companies should strongly consider providing treatments via compassionate use rather than patient assistance when requested treatments are to be used off indication.
When patients have limited treatment options, physicians and research teams working in academic medical centers, hospitals or community practices should help them find compassionate use opportunities. When biopharmaceutical companies approve compassionate use requests, it is important for physicians and research teams to report back on how patients are doing. While serious adverse event reporting is important, data on patient outcomes can be even more meaningful.
Finally, patients (or caregivers) should seek care with physicians who understand and are willing to prepare and submit compassionate use requests. There is important information to be gained from treating real-world patients in a compassionate use setting. Success in the compassionate use space will help evolve the way we study investigational products from one where we look for effectiveness in large groups of patients to one where research can be driven by individual circumstances for the broader benefit of patients.
Download a copy of the Anova White Paper ‘The Real World Value of Compassionate Use‘ here.
